JP2016027807A5 - - Google Patents

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JP2016027807A5
JP2016027807A5 JP2015176407A JP2015176407A JP2016027807A5 JP 2016027807 A5 JP2016027807 A5 JP 2016027807A5 JP 2015176407 A JP2015176407 A JP 2015176407A JP 2015176407 A JP2015176407 A JP 2015176407A JP 2016027807 A5 JP2016027807 A5 JP 2016027807A5
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標的DNAに特異的なガイドRNAもしくはガイドRNAをコードするDNA、およびCas9タンパク質コード核酸もしくはCas9タンパク質を含有する、真核細胞または真核生物中の標的DNAを切断するための組成物であって、ガイドRNAが標的DNAとハイブリダイズするcrRNA（CRISPR RNA）およびtracrRNA（トランス活性化crRNA）を含むデュアルRNA、またはcrRNAの一部およびtracrRNAの一部を含み、かつ標的DNAとハイブリダイズする一本鎖RNA（sgRNA）であり、標的DNAが真核細胞のDNAである、上記組成物。 DNA encoding a specific guide RNA or guide RNA to the target DNA, and containing Cas 9 protein encoding nucleic acid or Cas 9 protein, met composition for cleaving the target DNA of a eukaryotic cell or a eukaryotic organism Dual RNA containing crRNA (CRISPR RNA) and tracrRNA (trans-activated crRNA) that hybridize with target DNA, or part of crRNA and part of tracrRNA, and one that hybridizes with target DNA The above composition, which is a single-stranded RNA (sgRNA) and the target DNA is eukaryotic DNA . 標的DNAがcrRNAまたはsgRNAに相補的な配列を有するヌクレオチドを含む鎖、およびプロトスペーサー隣接モチーフ（PAM）を含む鎖を含む、請求項１に記載の組成物。The composition of claim 1, wherein the target DNA comprises a strand comprising a nucleotide having a sequence complementary to crRNA or sgRNA and a strand comprising a protospacer adjacent motif (PAM). crRNAまたはsgRNAが標的DNAに相補的な配列を有する20ヌクレオチドを含む、請求項１に記載の組成物。The composition of claim 1, wherein the crRNA or sgRNA comprises 20 nucleotides having a sequence complementary to the target DNA. Cas9タンパク質がそのC-またはN-末端に核局在化シグナル（NLS）を更に含む、請求項１に記載の組成物。The composition of claim 1, wherein the Cas9 protein further comprises a nuclear localization signal (NLS) at its C- or N-terminus. 真核細胞が哺乳類細胞である、請求項４に記載の組成物。The composition according to claim 4, wherein the eukaryotic cell is a mammalian cell. 標的DNAが真核細胞の内在性DNAである、請求項１に記載の組成物。 Target DNA is endogenous D NA eukaryotic composition of claim 1. Cas9タンパク質コード核酸またはCas9タンパク質がストレプトコッカス属菌（genus Streptococcus）に由来する、請求項１に記載の組成物。 The composition according to claim 1, wherein the Cas 9 protein-encoding nucleic acid or the Cas 9 protein is derived from a genus Streptococcus . 前記ストレプトコッカス属菌（genus Streptococcus）が化膿性連鎖球菌（Streptococcus pyogenes）である、請求項７に記載の組成物。 The Streptococcus genus (genus Streptococcus) is Streptococcus pyogenes (Streptococcus pyogen e s), The composition of claim 7. PAMが5'-NGG-3'のトリヌクレオチドである、請求項２に記載の組成物。The composition of claim 2, wherein the PAM is a 5′-NGG-3 ′ trinucleotide. 標的DNAの切断が部位特異的DNA二本鎖切断である、請求項１〜９のいずれか１項に記載の組成物。The composition according to any one of claims 1 to 9, wherein the cleavage of the target DNA is a site-specific DNA double-strand break. 請求項１〜９のいずれか１項に記載の組成物を含む、真核細胞または真核生物中の標的DNAを切断するためのキット。 A kit for cleaving a target DNA in a eukaryotic cell or a eukaryotic organism, comprising the composition according to any one of claims 1 to 9 . 単離された真核細胞または非ヒト真核生物にCas9タンパク質コード核酸もしくはCas9タンパク質、およびガイドRNAもしくはガイドRNAをコードするDNAを同時トランスフェクトまたは連続トランスフェクトするステップを含む、Cas9タンパク質およびガイドRNAを有する単離された真核細胞あるいは非ヒト真核生物を作製する方法であって、ガイドRNAが標的DNAとハイブリダイズするcrRNA（CRISPR RNA）およびtracrRNA（トランス活性化crRNA）を含むデュアルRNA、またはcrRNAの一部およびtracrRNAの一部を含み、かつ標的DNAとハイブリダイズする一本鎖RNA（sgRNA）であり、標的DNAが真核細胞のDNAである、上記方法。 Isolated eukaryotic cells or non-human eukaryotic organisms Cas 9 protein encoding nucleic acid or Cas 9 proteins, and guide comprising RNA or guide step RNA is cotransfected or continuous transfected with DNA encoding, Cas 9 protein And an isolated eukaryotic cell or non-human eukaryotic organism having a guide RNA, the method comprising a crRNA (CRISPR RNA) and a tracrRNA (transactivated crRNA) in which the guide RNA hybridizes with a target DNA The above method, wherein the RNA is a single-stranded RNA (sgRNA) that contains dual RNA or part of crRNA and part of tracrRNA and hybridizes with target DNA, and the target DNA is eukaryotic DNA . 標的DNAがcrRNAまたはsgRNAに相補的な配列を有するヌクレオチドを含む鎖、およびプロトスペーサー隣接モチーフ（PAM）を含む鎖を含む、請求項１２に記載の方法。13. The method of claim 12, wherein the target DNA comprises a strand comprising a nucleotide having a sequence complementary to crRNA or sgRNA, and a strand comprising a protospacer adjacent motif (PAM). crRNAまたはsgRNAが標的DNAに相補的な配列を有する20ヌクレオチドを含む、請求項１２に記載の方法。13. A method according to claim 12, wherein the crRNA or sgRNA comprises 20 nucleotides having a sequence complementary to the target DNA. 真核細胞が哺乳類細胞である、請求項１２に記載の方法。The method according to claim 12, wherein the eukaryotic cell is a mammalian cell. 標的DNAが真核細胞の内在性DNAである、請求項１２に記載の方法。13. The method according to claim 12, wherein the target DNA is eukaryotic endogenous DNA. Cas9タンパク質コード核酸またはCas9タンパク質がストレプトコッカス属菌（genus Streptococcus）に由来する、請求項１２に記載の方法。 The method according to claim 12 , wherein the Cas 9 protein-encoding nucleic acid or the Cas 9 protein is derived from a genus Streptococcus . 前記ストレプトコッカス属菌（genus Streptococcus）が化膿性連鎖球菌（Streptococcus pyogenes）である、請求項１７に記載の方法。 The Streptococcus genus (genus Streptococcus) is Streptococcus pyogenes (Streptococcus pyogen e s), The method of claim 17. PAMが5'-NGG-3'のトリヌクレオチドである、請求項１３に記載の方法。14. The method of claim 13, wherein the PAM is a 5'-NGG-3 'trinucleotide. トランスフェクトするステップが、最初にCas9タンパク質コード核酸によるトランスフェクション、続いてガイドRNAによるトランスフェクションを含む連続トランスフェクションによって行われる、請求項１２に記載の方法。13. The method of claim 12, wherein the step of transfecting is performed by sequential transfection comprising first transfection with a Cas9 protein-encoding nucleic acid followed by transfection with a guide RNA. Cas9タンパク質がそのC-またはN-末端に核局在化シグナル（NLS）を更に含む、請求項１２〜２０のいずれか１項に記載の方法。21. The method of any one of claims 12-20, wherein the Cas9 protein further comprises a nuclear localization signal (NLS) at its C- or N-terminus. 標的DNAを有する単離された真核細胞または非ヒト真核生物に請求項１〜９のいずれか１項に記載の組成物をトランスフェクトするステップを含む、単離された真核細胞または非ヒト真核生物中の標的DNAを切断する方法。 Comprising the step of transfecting a composition according to any one of claims 1 to 9, eukaryotic cells or non-human eukaryotic molecule which has a target DNA, isolated eukaryotic cells or non A method for cleaving target DNA in human eukaryotes. 真核生物が非ヒト哺乳類または植物である、請求項２２に記載の方法。 23. The method of claim 22 , wherein the eukaryote is a non-human mammal or plant. トランスフェクトするステップが同時トランスフェクションまたは連続トランスフェクションによって行われる、請求項２２に記載の方法。 24. The method of claim 22 , wherein the transfection step is performed by co-transfection or sequential transfection. トランスフェクトするステップが、最初にCas9タンパク質コード核酸によるトランスフェクション、続いてガイドRNAによるトランスフェクションを含む連続トランスフェクションによって行われる、請求項２４に記載の方法。 Step transfecting is first transfection with Cas 9 protein encoding nucleic acid is subsequently carried out by continuously transfection including by belt transfection the guide RNA, The method of claim 24. 標的DNAの切断が部位特異的DNA二本鎖切断である、請求項２２に記載の方法。The method according to claim 22, wherein the cleavage of the target DNA is a site-specific DNA double-strand break. 請求項１〜９のいずれか１項に記載の組成物によって編集されたゲノムを有する非ヒト動物の胚。 A non-human animal embryo having a genome edited by the composition according to any one of claims 1 to 9 . 請求項２７に記載の胚を非ヒト動物の卵管に移入することによって得られるゲノム改変非ヒト動物。 A genome-modified non-human animal obtained by transferring the embryo according to claim 27 into a fallopian tube of a non-human animal. 請求項１〜９のいずれか１項に記載の組成物を非ヒト動物の胚に導入するステップ；およびその胚を偽妊娠した代理母の卵管に移入して、ゲノム改変非ヒト動物を生じさせるステップ、を含む、ゲノム改変非ヒト動物を作製する方法であって、偽妊娠した代理母が非ヒト動物である、上記方法。 Step introducing a composition as described in the non-human animal embryo to any one of claims 1-9; and transferred into the oviduct of a surrogate mother to and the embryos were pseudopregnant, resulting genomic altered non-human animal A method for producing a genome-modified non-human animal, wherein the pseudopregnant surrogate mother is a non-human animal . 以下：Less than:
Cas9タンパク質コード核酸もしくはCas9タンパク質；およびCas9 protein-encoding nucleic acid or Cas9 protein; and
標的DNAに特異的なガイドRNAもしくはガイドRNAをコードするDNAGuide RNA specific to target DNA or DNA encoding guide RNA
を有し、ガイドRNAが標的DNAとハイブリダイズするcrRNA（CRISPR RNA）およびtracrRNA（トランス活性化crRNA）を含むデュアルRNA、またはcrRNAの一部およびtracrRNAの一部を含み、かつ標的DNAとハイブリダイズする一本鎖RNA（sgRNA）である、単離された真核細胞または非ヒト真核生物。Dual RNA containing crRNA (CRISPR RNA) and tracrRNA (trans-activated crRNA) that hybridize with target DNA, or part of crRNA and part of tracrRNA, and hybridizes with target DNA An isolated eukaryotic cell or non-human eukaryote that is a single stranded RNA (sgRNA). 標的DNAがcrRNAまたはsgRNAに相補的な配列を有するヌクレオチドを含む鎖、およびプロトスペーサー隣接モチーフ（PAM）を含む鎖を含む、請求項３０に記載の単離された真核細胞または非ヒト真核生物。31. The isolated or non-human eukaryotic cell of claim 30, wherein the target DNA comprises a strand comprising a nucleotide having a sequence complementary to crRNA or sgRNA, and a strand comprising a protospacer adjacent motif (PAM). Creatures. crRNAまたはsgRNAが標的DNAに相補的な配列を有する20ヌクレオチドを含む、請求項３０に記載の単離された真核細胞または非ヒト真核生物。31. The isolated eukaryotic cell or non-human eukaryote of claim 30, wherein crRNA or sgRNA comprises 20 nucleotides having a sequence complementary to the target DNA. 真核細胞が哺乳類細胞である、請求項３０に記載の単離された真核細胞または非ヒト真核生物。31. The isolated eukaryotic cell or non-human eukaryotic organism according to claim 30, wherein the eukaryotic cell is a mammalian cell. 標的DNAが真核細胞の内在性DNAである、請求項３０に記載の単離された真核細胞または非ヒト真核生物。31. The isolated or non-human eukaryote of claim 30, wherein the target DNA is an eukaryotic endogenous DNA. Cas9タンパク質コード核酸またはCas9タンパク質がストレプトコッカス属菌（genus Streptococcus）に由来する、請求項３０に記載の単離された真核細胞または非ヒト真核生物。31. The isolated eukaryotic cell or non-human eukaryotic organism of claim 30, wherein the Cas9 protein-encoding nucleic acid or Cas9 protein is derived from a genus Streptococcus. 前記ストレプトコッカス属菌（genus Streptococcus）が化膿性連鎖球菌（Streptococcus pyogenes）である、請求項３５に記載の単離された真核細胞または非ヒト真核生物。36. The isolated eukaryotic cell or non-human eukaryote of claim 35, wherein the genus Streptococcus is Streptococcus pyogenes. PAMが5'-NGG-3'のトリヌクレオチドである、請求項３０に記載の単離された真核細胞または非ヒト真核生物。31. The isolated eukaryotic or non-human eukaryote of claim 30, wherein the PAM is a 5'-NGG-3 'trinucleotide. Cas9タンパク質がそのC-またはN-末端に核局在化シグナル（NLS）を更に含む、請求項３０〜３７のいずれか１項に記載の単離された真核細胞または非ヒト真核生物。38. The isolated eukaryotic cell or non-human eukaryotic organism according to any one of claims 30 to 37, wherein the Cas9 protein further comprises a nuclear localization signal (NLS) at its C- or N-terminus.
JP2015176407A 2012-10-23 2015-09-08 Composition for cleaving target dna comprising guide rna specific for target dna and cas protein-encoding nucleic acid or cas protein, and use thereof Pending JP2016027807A (en)

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